Epstein Leonard H, Dallery Jesse
Jacobs School of Medicine and Biomedical Sciences, Division of Behavioral Medicine, Department of Pediatrics, University at Buffalo, Buffalo, New York, United States of America.
Department of Psychology, University of Florida, Gainesville, Florida, United States of America.
Harv Data Sci Rev. 2022;4(SI3). doi: 10.1162/99608f92.ff9300a8. Epub 2022 Sep 8.
Single-case experimental designs (SCEDs) represent a family of research designs that use experimental methods to study the effects of treatments on outcomes. The fundamental unit of analysis is the single case-which can be an individual, clinic, or community-ideally with replications of effects within and/or between cases. These designs are flexible and cost-effective and can be used for treatment development, translational research, personalized interventions, and the study of rare diseases and disorders. This article provides a broad overview of the family of single-case experimental designs with corresponding examples, including reversal designs, multiple baseline designs, combined multiple baseline/reversal designs, and integration of single-case designs to identify optimal treatments for individuals into larger randomized controlled trials (RCTs). Personalized N-of-1 trials can be considered a subcategory of SCEDs that overlaps with reversal designs. Relevant issues for each type of design-including comparisons of treatments, design issues such as randomization and blinding, standards for designs, and statistical approaches to complement visual inspection of single-case experimental designs-are also discussed.
单病例实验设计(SCEDs)是一类研究设计,其使用实验方法来研究治疗对结果的影响。基本分析单位是单个病例——可以是个体、诊所或社区——理想情况下,病例内部和/或病例之间的效应具有可重复性。这些设计灵活且具有成本效益,可用于治疗开发、转化研究、个性化干预以及罕见疾病和病症的研究。本文对单病例实验设计家族进行了广泛概述,并给出了相应示例,包括反转设计、多基线设计以及多基线/反转组合设计,还介绍了如何将单病例设计整合到更大规模的随机对照试验(RCTs)中以确定个体的最佳治疗方案。个性化N-of-1试验可被视为与反转设计重叠的单病例实验设计的一个子类别。还讨论了每种设计类型的相关问题,包括治疗比较、随机化和盲法等设计问题、设计标准以及补充单病例实验设计视觉检查的统计方法。